Cystic Fibrosis and Stem Cell Therapy


Cystic fibrosis is a genetic disease that leads to the destruction and damage to the digestive system. It also affects other organs of the body, such as the renal system, liver, and the intestine. Chronic symptoms of this disease include pulmonary infections, difficulty breathing, and the productive cough due to the infection of the lungs by microorganisms. Other signs and symptoms of this disease include fatty steatorrhea, infertility in males. People often present with different kinds of symptoms. This disease can be inherited from one’s parents. It’s an autosomal recessive genetic disease. It’s due to a defective gene, known as the CTFR gene. Cystic fibrosis has no cure, however, treatments should be given to the patients to improve their symptoms, and to also make life more comfortable for them.

What Are the Signs and Symptoms Of Cystic Fibrosis?

The most common signs and symptoms of cystic fibrosis is skin that tastes like salt, associated with poor growth, inadequate weight gain, even though they eat well, and regularly. This disease is tested for in every state of America. It can be successfully diagnosed in the first month of life of a fetus, even before symptoms start presenting. The signs and symptoms of this disease vary in people, in fact, symptoms may deteriorate in the same patient as time passes by. On the other hand, some affected people might not experience any symptom at all. Apart from their skin tasting like salt, it also has a high amount of salt (more than the normal amount of salt). The guardians or parents of affected kids can taste the salt when playing around with their kids, or just kissing them. Adults affected by this disease usually presents with symptoms that are different from kids. The disease mostly affects the organs of the abdomen such as the pancreas, and can also lead to infertility and pulmonary diseases.

  1. Pulmonary system: Patients affected by cystic fibrosis often present with thick mucus. This thick mucus does cause the obstruction of the lungs, preventing the free flow of air in the respiratory system. Below are some of the symptoms that could be presented when the pulmonary system is affected;

  2. Wheezing

  3. Productive cough that is persistent and with thick mucus

  4. Frequent infection of the lungs.

  5. Inflammation of the nasal canals

  6. Stuffy nose, due to the thick mucus

  7. Breathlessness

  8. Patients affected with cystic fibrosis becomes tired and lethargic on exertion. They usually cannot tolerate exercise.

  9. Digestive system: The thick mucus that is produced due to cystic fibrosis does block the tubes of the digestive system. They prevent the transport of enzymes from the pancreas to the duodenum. The body cannot properly digest and absorb food materials into the bloodstream. This would eventually lead to the following;

  10. Steatorrhea: This means the production of fatty and foul-smelling The stool also appears greasy due to the high level of fat in it.

  11. Patients do have poor weight gain and retarded growth

  12. The thick mucus produced may also block the intestine, especially in newborns.

  13. It can also lead to constipation.

The difficulty of passing out stools in cystic fibrosis can lead to the outward protrusion of the rectum. This is known as rectal prolapse. This is an indication of cystic fibrosis when it happens in kids. Rectal prolapse in kids may have to be treated through surgical procedures.

  1. Endocrine: Cystic fibrosis can induce the damage of some endocrine glands, such as the pancreas. The islet of Langerhans of the pancreas is responsible for the production of insulin. The damage of the pancreas would lead to the reduction or total stop of the production and release of insulin in the body. Insulin is needed for the digestion of glucose. This condition might eventually lead to the development of type 1 and type 2 diabetes mellitus. In addition, there are some fat-soluble vitamins, that cannot be absorbed without the fat enzymes of the pancreas. An example of such vitamin is Vitamin D. The malabsorption of Vitamin from food would lead to bone diseases such as rickets in kids and osteoporosis in adults.

  2. Infertility: This occurs in both men and women. According to statistics, about 97% of men affected with cystic fibrosis are infertile. However, they’re not sterile and are capable of having children with the many available assisted reproductive techniques. The major cause of infertility in men is the absence of the vas deferens, which is usually absent from birth. On the other hand, about twenty percent of women affected by cystic fibrosis do have infertility. In severe cases of cystic fibrosis, women might have problems with ovulation and can also lack menstruation entirely.

What Are the Causes Of Cystic Fibrosis?

Cystic fibrosis is caused by a defect in a gene, called the CFTR gene. This gene is responsible for the control and regulation of the movement of salt in the cells. However, this gene defects in cystic fibrosis, and affected individuals present with thick mucus in many systems of the body, such as the gastrointestinal system, pulmonary and the reproductive system. It’s a genetic disease, and also an autosomal recessive disease. This means that children would have to inherit a copy of the gene from both parents to have the disease.

How Is Cystic Fibrosis Currently Treated?

There is no cure for cystic fibrosis, however, treatments can be given to improving the symptoms, and make life more comfortable for the patients. Cystic fibrosis is a complex disease to manage. Below are some of the treatment given;

  1. Antibiotics are administered to protect and the patient from lung infections and to also treat it.

  2. Medications such as bronchodilators are also given to keep the patient’s airways open.

  3. Enzymes

Stem Cell Therapy For Cystic Fibrosis

Cystic fibrosis is a good candidate for stem cell therapy because it’s caused by a defect in the genetic makeup. Stem cells such as the hematopoietic and mesenchymal stem cells have proved to be useful in the treatment of this. In some studies, stem cells have been introduced into the lungs, and they’ve developed and differentiated to form new cells of the respiratory system. Replacing the cells damaged as a result of the disease. However, more research and studies are still being done. Stem cell therapy is being relied on to be the permanent cure for cystic fibrosis in the nearest future.

References

119 Insulin Glargine (IG) for therapy of Cystic Fibrosis Related Diabetes (CFRD) and Impaired Glucose Tolerance (IGT) in Cystic Fibrosis (CF) patients (pts). (2005). Journal of Cystic Fibrosis, 4, p.S32.

277 Cystic Fibrosis in adults. (2005). Journal of Cystic Fibrosis, 4, p.S73.

Conese, M. and Rejman, J. (2006). Stem cells and cystic fibrosis. Journal of Cystic Fibrosis, 5(3), pp.141-143.

Wang, G. (2017). New Frontiers in Cystic Fibrosis Therapy: The Case of Stem Cells. Clinical Immunology, E

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